With the advancement of research on rare ocular diseases such as inherited retinal dystrophy (IRD) has advanced in recent years, especially breakthroughs in therapeutic approaches represented by cell and gene therapy, potential intervention strategies have emerged for these conditions. Establishing standardized endpoints and evaluation methods for visual function in patients with IRD has become crucial for assessing disease progression, safety, and therapeutic efficacy of innovative treatments. Best corrected visual acuity (BCVA) is widely recognized as one of the primary endpoints for assessing visual function. However, for IRD patients with severe rod photoreceptor dysfunction, who often present with profound low vision or even legal blindness, the applicability of BCVA as a traditional visual function indicator is limited. The multi-luminance mobility test (MLMT) has emerged as a functional visual assessment tool that evaluates the ability to navigate obstacle courses under varying illumination levels. By establishing graded evaluation standards, MLMT objectively quantifies the impact of lighting conditions on patients’ nobility and spatial orientation, providing a novel quantitative tool for assessing visual function in IRD clinical trials. Currently, there is a lack of unified and standardized guidelines for the use of MLMT in China, posing challenges to its implementation in practical clinical research. To address this, Ocular Fundus Disease Society of Chinese Medical Association and Chinese Medical Doctor Association convened a multidisciplinary team comprising clinical experts in genetic retinal diseases, statisticians, and optical specialists to investigate the current applications and technical characteristics of MLMT, ultimately formulating consensus recommendations for its use as a clinical trial endpoint for IRD gene therapies. This consensus aims to provide a set of MLMT operating norms applicable to China's national conditions, guide clinical practice and research in ophthalmology and related disciplines, and promote the standardization process of IRD clinical trials in China, so as to better serve the IRD patient population and promote the development of related fields.
Objective To investigate and analyze the incidence and clinical characteristics of malnutrition in patients with tuberculosis and type 2 diabetes. Methods four hundred patients with tuberculosis and type 2 diabetes were diagnosed in the tuberculosis Department of West China Hospital of Sichuan University from June 1, 2018 to April 30, 2023. The incidence and clinical characteristics of malnutrition were analyzed using the nutritional risk Screening 2002 score and the Global Malnutrition Leadership Initiative criteria. Results 170 patients (42.5%) were malnourished. In malnourished patients, 78 cases (45.9%) had fever, 132 cases (77.6%) had cough, 44 cases (25.9%) had hemoptysis, and 36 cases (21.2%) had night sweating. Compared with the non-malnourished patients, the difference was statistically significant (P<0.05). Lymphocyte count was (1.0±0.5)×109/L, albumin was (31.4±20.5)g/L; CD3 count 792.7±205.0, CD4 count 535.4±15.0, CD8 count 429.5±123.0, CD3%: 65.5%±11.1%, CD4%: 39.1%±9.6%, CD8%: 19.1%±9.6%, CD4/CD8: 1.3±0.2, which were significantly lower than those in the group without malnutrition. Pulmonary cavity occurred in 44 cases (25.9%), lesions in 2 - 4 lung fields in 76 cases (44.7%) and lesions in ≥5 lung fields in 52 cases (30.6%), all of which were significantly higher than those in the non-malnutrition group. The above differences were statistically significant.Conclusion We should pay more attention to the screening of malnutrition in patients with tuberculosis and type 2 diabetes. Patients with malnutrition have more obvious clinical symptoms, lower immunity, and more serious imaging lesions.
Duchenne muscular dystrophy is an X-linked inherited progressive degenerative muscle disease caused by mutations in the dystrophin gene, and is one of the most common progressive muscular dystrophies. We will review the selection of genetic diagnosis methods for Duchenne muscular dystrophy, the selection of experimental animal models, and treatment for the primary cause (including gene replacement therapy, exon skipping therapy, genome editing, stop codon read-through therapy, and stem cell therapy), the treatment of secondary pathological reactions and methods of assessing disease progression. The purpose is to enrich clinicians’ knowledge of the disease and provide a reference and help for the clinical diagnosis and treatment of Duchenne muscular dystrophy.
Objective To evaluate the efficiency and security of physical exercise with low intensity against malnutrition and sarcopenia in patients with cirrhosis. Methods Between December 2014 and October 2015, 37 patients with cirrhosis were divided into two groups according to their willings, with 19 in the exercise group and 18 in the control group. Endurance of the exercise for 3 months were recorded. Mid-arm circumference, " up and go” time, width of portal vein and Child-Pugh score were compared before and after the research between the two groups. Results Three months later, the mid-arm circumference and the " up and go” time of the exercise group [(33.99±2.15) cm, (9.17±0.35) s] were better than those before the exercise [(32.09±2.58) cm, (9.77±0.46) s] and those in the control group [(31.93±2.04) cm, (9.76±0.30) s], and the differences above were all statistically significant (P<0.05). The change of the width of portal vein was positively correlated with pre-exercise body mass index in overweight patients (r=0.93, P=0.007). Conclusions Physical exercise with low intensity is safe and effective against malnutrition and sarcopenia in patients with cirrhosis. Overweitht patiens or malnutrition at the early stage may benefit more.
ObjectiveTo evaluate the impact of using alanyl-glutamine dipeptide on clinical outcome for gastric cancer patients with nutritional risk after total gastrectomy. MethodsThis study was carried out in the period from March to August 2015. The nutritional risk was screened by continuous sampling method in the new hospitalized patients with gastric cancer who would undergo total gastrectomy. The patients were grouped randomly. Alanyl-glutamine was given to the experimental group patients. The clinical data of the two groups were analyzed, such as the laboratory parame-ters of nutritional status and hepatorenal function, complications of surgery, the nutrition-related hospitalization day, etc. ResultsThe preoperative data were consistent in the two groups of the included 40 cases. The results showed, in the third and seventh days after surgery, the level of plasma albumin was higher in the experimental group than in the control group〔(33.9±5.6) g/L vs. (30.8±4.0) g/L and (36.6±3.9) g/L vs. (33.9±4.2) g/L, respectively). Also, the CD4+/CD8+ cells immune index was significantly improved in the experimental group after surgery (1.7±0.7 vs. 1.2±0.3, P < 0.05). The recovery time of intestinal function〔(65.7±5.3) h vs. (71.6±7.2)h, P < 0.01)〕and nutrition-related hospitalization day〔(10.1±1.8) d vs. (11.7±1.9)d, P < 0.01)〕in alanyl-glutamine dipeptide group were shorted than that in the control group. No serious adverse drug reactions were found in the patients during the treatment period. ConclusionApplication alanyl-glutamine to the patients with nutritional risk after total gastrectomy could partly improve clinical outcome indicators.
【摘要】 目的 探索持续非卧床腹膜透析（CAPD）患者年龄与营养状况的关系，为营养教育提供指导。 方法 对2007年8月-2010年1月腹膜透析177例患者的临床资料进行回顾性分析，包括血红蛋白（HGB）、血清白蛋白（ALB）、年龄、体重指数（BMI）、腹膜平衡试验（PET）、尿素清除指数（Kt/V）、24 h尿蛋白、腹透液漏出蛋白、标准化每日蛋白质分解率（nPCR）等，分析年龄与营养状况的关系。 结果 老年组（≥60岁）和中青年组（lt;60岁）ALB分别为（31.54±5.91）、（33.24±4.75）g/L，老年组ALB较低（Plt;0.05）；老年组Kt/V值1.95±0.63和HGB水平（95.25±22.89） g/L均高于青年组（Plt;0.05）。两组患者营养不良与BMI、PET、每日蛋白漏出总量、nPCR无关。 结论 CAPD老年患者（≥60岁）更容易发生营养不良，且老年组低蛋白血症与透析充分性无明显关系。故应加强老年CAPD患者的饮食营养管理指导，设计合理的饮食。【Abstract】 Objective To investigate the relationship between age and nutrition status of the patients undergoing continuous ambulatory peritoneal dialysis (CAPD), and provide instructions for nutrition education. Methods We retrospectively analyzed the clinical data of 177 patients having undergone CAPD in the Department of Nephrology of West China Hospital between April 2007 and January 2010. Clinical parameters collected included hemoglobin (HGB), serum albumin (ALB), age, body mass index (BMI), results of the peritoneal equilibrium test (PET), urea clearance index (Kt/V), 24-hour urinary protein, protein loss in the peritoneal dialysate, normalized protein catabolic rate (nPCR). Relationship between age and nutrition status in these patients were analyzed. Results Plasma albumin was (31.54±5.91) g/L and (33.24±4.75) g/L respectively in patients of or older than 60 years old (the elderly group) and in patients younger than 60 years old (the younger group). Compared with the younger group, patients in the elderly group had lower ALB (Plt;0.05), but higher Kt/V 1.95±0.63 and hemoglobin level (95.25±22.89) g/L (Plt;0.05). In addition, malnutrition in both groups was not associated with BMI, PET results, daily protein loss in the peritoneal dialysate or nPCR. Conclusions Patients of or older than 60 years old undergoing CAPD are more likely to develop malnutrition, indicating that hypoalbuminemia is not associated with dialysis inadequacy. We advocate enhancing diet nutrition education in elderly CAPD patients and providing them with balanced diet regime.
Objective To summarize the nutritional assessment methods for liver cancer patients and their development, and to provide reference for rationally nutritional assessment and nutritional support. Method Domestic and foreign literatures were searched to summarize the nutritional assessment methods for liver cancer patients and their development, in order to determine a practical and feasible assessment method. Results The evaluation validity of traditionally nutritional assessment methods which contained many individual indicators was low. But subjective global assessment (SGA), mini nutritional assessment (MNA), and nutritional risk screening 2002 (NRS-2002) had similar evaluation validity, this 3 kinds of nutritional assessment methods were more suitable for liver cancer patients compared with the traditionally nutritional assessment methods. Conclusion The clinician should simultaneously apply SGA, MNA, NRS-2002, and other comprehensively nutritional assessment methods, as well as related anthropometric and laboratory indexes, to get a more accurate assessment of the nutritional status for patients with liver cancer.
ObjectiveTo explore the diagnostic efficacy of Geriatric Nutritional Risk Index (GNRI) in malnutrition of elderly patients with chronic obstructive pulmonary disease (COPD) in outpatient department. MethodsOne hundred and five elderly outpatients with COPD were enrolled in the study, and their nutritional screening was carried out. The clinical and laboratory parameters of patients in the normal nutrition group (high GNRI group) and malnutrition group (low GNRI group) were compared, and the correlation analysis was conducted. The diagnostic efficacy of GNRI was evaluated based on the malnutrition universal screening tool (MUST). ResultsThe prevalence of malnutrition was high in COPD elderly outpatients. The prevalence of malnutrition in group D was 61.8%. There were significant differences between the two groups in body mass index, serum albumin, FEV1 percentage in the predicted value, 6-minute walk distance, and the number of acute exacerbations in the past year. GNRI was significantly related to the above parameters. The sensitivity, specificity and accuracy of GNRI were 81.8%, 83.6% and 82.9%, using MUST as the standard. ConclusionGNRI can be used for nutritional screening of COPD patients in elderly outpatients, which is simple, convenient and relatively accurate, and can be popularized in other medical institutions.